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from the YADIDNKE-Yet-Another-Disease-I-Did-Not-Know-Existed dept.
Promising Results with Givosiran in Acute Hepatic Porphyrias[*]
Investigational givosiran met the primary endpoint of reduction in the annualized rate of composite porphyria attacks versus placebo in patients with acute hepatic porphyria (AHP), researchers said here.
In the interim analysis of the phase III ENVISION trial, patients treated with givosiran experienced a mean composite annualized rate of 3.2 attacks versus 12.5 attacks in patients on placebo (P<0.0001), for a mean reduction of 74%, said Manisha Balwani, MD, of the Mount Sinai School of Medicine in New York City, and colleagues.
"We saw a robust treatment effect," said Balwani at a press conference at the European Association for the Study of the Liver (EASL) annual meeting.
"Currently, there are no approved therapies aimed at preventing the painful, often incapacitating attacks, and chronic symptoms associated with acute hepatic porphyria," she added. "The results from ENVISION are promising and demonstrate a strong treatment effect for givosiran, with reduction of attacks and improvement in patient-reported measures of overall health status and quality of life."
[*] Porphyria.
BBC has a dumbed down article: Gene-silencing: 'New class' of medicine reverses disease porphyria
(Score: 0) by Anonymous Coward on Tuesday April 16 2019, @05:17PM
The primary outcome measures had to be declared before starting the trial. I've only heard of one or two cases where the primary outcome measures have been updated mid-trial and they need a very good reason why the new outcome measure is better.
Also, any analysis done before the trial is completed will be "interim" regardless of the fact that the data will not change (there will not be any additional patients or changes to the primary outcome measure).